SI2497500T1 - Gene therapy for spinal muscular atrophy - Google Patents

Gene therapy for spinal muscular atrophy

Info

Publication number: SI2497500T1
Authority: SI; Slovenia
Prior art keywords: gene therapy; muscular atrophy; spinal muscular; spinal; atrophy
Prior art date: 2006-10-03

Application number

SI200731873A

Other languages

Slovenian (sl)

Inventor

Catherine O'riordan

Samuel Wadsworth

Original Assignee

Genzyme Corporation

Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)

2006-10-03

Filing date

2007-10-03

Publication date

2017-01-31

2007-10-03 Application filed by Genzyme Corporation filed Critical Genzyme Corporation

2017-01-31 Publication of SI2497500T1 publication Critical patent/SI2497500T1/en

Classifications

- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14145—Special targeting system for viral vectors
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2810/00—Vectors comprising a targeting moiety
- C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
- C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
- C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
- C12N2830/002—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination inducible enhancer/promoter combination, e.g. hypoxia, iron, transcription factor
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination

Landscapes

Health & Medical Sciences (AREA)
Life Sciences & Earth Sciences (AREA)
Engineering & Computer Science (AREA)
Genetics & Genomics (AREA)
Chemical & Material Sciences (AREA)
Bioinformatics & Cheminformatics (AREA)
General Health & Medical Sciences (AREA)
Biotechnology (AREA)
Zoology (AREA)
Veterinary Medicine (AREA)
Medicinal Chemistry (AREA)
Organic Chemistry (AREA)
Pharmacology & Pharmacy (AREA)
Public Health (AREA)
Animal Behavior & Ethology (AREA)
Biomedical Technology (AREA)
Wood Science & Technology (AREA)
General Engineering & Computer Science (AREA)
Epidemiology (AREA)
Molecular Biology (AREA)
Neurology (AREA)
Immunology (AREA)
Proteomics, Peptides & Aminoacids (AREA)
Microbiology (AREA)
Gastroenterology & Hepatology (AREA)
Biochemistry (AREA)
Virology (AREA)
Physics & Mathematics (AREA)
Biophysics (AREA)
Marine Sciences & Fisheries (AREA)
Plant Pathology (AREA)
Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
General Chemical & Material Sciences (AREA)
Chemical Kinetics & Catalysis (AREA)
Neurosurgery (AREA)
Physical Education & Sports Medicine (AREA)
Orthopedic Medicine & Surgery (AREA)
Psychiatry (AREA)
Hospice & Palliative Care (AREA)
Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

SI200731873A 2006-10-03 2007-10-03 Gene therapy for spinal muscular atrophy SI2497500T1 (en)

Applications Claiming Priority (2)

Application Number	Priority Date	Filing Date	Title
US82797706P	2006-10-03	2006-10-03
EP12157957.7A EP2497500B1 (en)	2006-10-03	2007-10-03	Gene therapy for spinal muscular atrophy

Publications (1)

Publication Number	Publication Date
SI2497500T1 true SI2497500T1 (en)	2017-01-31

Family

ID=39269037

Family Applications (3)

Application Number	Title	Priority Date	Filing Date
SI200731873A SI2497500T1 (en)	2006-10-03	2007-10-03	Gene therapy for spinal muscular atrophy
SI200731083T SI2066791T1 (en)	2006-10-03	2007-10-03	Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
SI200732128T SI3146982T1 (en)	2006-10-03	2007-10-03	Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders

Family Applications After (2)

Application Number	Title	Priority Date	Filing Date
SI200731083T SI2066791T1 (en)	2006-10-03	2007-10-03	Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
SI200732128T SI3146982T1 (en)	2006-10-03	2007-10-03	Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders

Country Status (13)

Country	Link
US (2)	US9890394B2 (en)
EP (4)	EP3632472A1 (en)
CN (3)	CN108853517A (en)
AR (1)	AR063113A1 (en)
CY (1)	CY1118477T1 (en)
DK (2)	DK2497500T3 (en)
ES (3)	ES2607060T3 (en)
HU (2)	HUE031182T2 (en)
LT (2)	LT3146982T (en)
PL (2)	PL2497500T3 (en)
PT (3)	PT2497500T (en)
SI (3)	SI2497500T1 (en)
WO (1)	WO2008042420A2 (en)

Families Citing this family (20)

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SI2497500T1 (en)	2006-10-03	2017-01-31	Genzyme Corporation	Gene therapy for spinal muscular atrophy
US20110286972A1 (en) *	2008-01-16	2011-11-24	Katsuhito Takahashi	Virus growing in hypoxic cell or virus vector expressing gene therein
RS67077B1 (en)	2009-05-02	2025-08-29	Genzyme Corp	Gene therapy for neurodegenerative disorders
US9770471B2 (en)	2011-08-17	2017-09-26	President And Fellows Of Harvard College	Conversion of somatic cells into functional spinal motor neurons, and methods and uses thereof
KR102584655B1 (en)	2014-11-14	2023-10-06	보이저 테라퓨틱스, 인크.	Modulatory polynucleotides
MX2017006216A (en)	2014-11-14	2018-08-29	Voyager Therapeutics Inc	Compositions and methods of treating amyotrophic lateral sclerosis (als).
EP3250279B1 (en) *	2015-01-30	2020-08-19	The Regents of the University of California	Spinal subpial gene delivery system
CL2015003024A1 (en)	2015-10-09	2016-05-13	Univ Chile	Method of genetic treatment using aav-xbp1s / gfp virus, and its use in the prevention and treatment of amyotrophic lateral sclerosis.
JP6949867B2 (en)	2016-03-28	2021-10-13	ザリージェンツオブザユニバーシティオブカリフォルニアＴｈｅＲｅｇｅｎｔｓＯｆＴｈｅＵｎｉｖｅｒｓｉｔｙＯｆＣａｌｉｆｏｒｎｉａ	Methods and Compositions for Treating Nerve Hyperexcitability
RU2758488C2 (en)	2016-05-18	2021-10-28	Вояджер Терапьютикс, Инк.	Modulating polynucleotides
CA3044291A1 (en) *	2016-11-17	2018-05-24	Nationwide Children's Hospital Inc.	Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2
JOP20190200A1 (en)	2017-02-28	2019-08-27	Univ Pennsylvania	Compositions useful in treatment of spinal muscular atrophy
AU2018261790B2 (en)	2017-05-05	2024-10-03	Voyager Therapeutics, Inc.	Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
AU2018317807A1 (en) *	2017-08-16	2020-02-06	Roxiant ApS	VTFT isoform of a BPIFB4 protein for use in neuronal diseases and injuries
AU2018327253B2 (en)	2017-09-08	2024-05-16	The Regents Of The University Of California	Method and composition for treating neuropathic pain
US11434502B2 (en)	2017-10-16	2022-09-06	Voyager Therapeutics, Inc.	Treatment of amyotrophic lateral sclerosis (ALS)
EP4454654A3 (en)	2017-10-16	2025-02-19	Voyager Therapeutics, Inc.	Treatment of amyotrophic lateral sclerosis (als)
JP7373226B2 (en)	2019-01-10	2023-11-02	ザリージェンツオブザユニバーシティオブカリフォルニア	Subpial delivery system and method of use
USD963164S1 (en)	2020-01-09	2022-09-06	The Regents Of The University Of California	Surgical needle
EP4370679A4 (en) *	2021-07-16	2025-11-12	Harvard College	MOTOR NEURON EXPRESSION ENHANCERS

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US5672344A (en)	1987-12-30	1997-09-30	The Regents Of The University Of Michigan	Viral-mediated gene transfer system
JP3532566B2 (en)	1993-06-24	2004-05-31	エル．グラハム，フランク	Adenovirus vectors for gene therapy
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SI2497500T1 (en)	2006-10-03	2017-01-31	Genzyme Corporation	Gene therapy for spinal muscular atrophy
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2007
- 2007-10-03 SI SI200731873A patent/SI2497500T1/en unknown
- 2007-10-03 CN CN201810575782.XA patent/CN108853517A/en active Pending
- 2007-10-03 SI SI200731083T patent/SI2066791T1/en unknown
- 2007-10-03 PL PL12157957T patent/PL2497500T3/en unknown
- 2007-10-03 EP EP19192274.9A patent/EP3632472A1/en active Pending
- 2007-10-03 CN CN201610214446.3A patent/CN105770910A/en active Pending
- 2007-10-03 WO PCT/US2007/021272 patent/WO2008042420A2/en not_active Ceased
- 2007-10-03 LT LT16184966T patent/LT3146982T/en unknown
- 2007-10-03 DK DK12157957.7T patent/DK2497500T3/en active
- 2007-10-03 ES ES12157957.7T patent/ES2607060T3/en active Active
- 2007-10-03 ES ES07839216T patent/ES2391789T3/en active Active
- 2007-10-03 ES ES16184966T patent/ES2757723T3/en active Active
- 2007-10-03 HU HUE12157957A patent/HUE031182T2/en unknown
- 2007-10-03 SI SI200732128T patent/SI3146982T1/en unknown
- 2007-10-03 EP EP07839216A patent/EP2066791B1/en active Active
- 2007-10-03 LT LTEP12157957.7T patent/LT2497500T/en unknown
- 2007-10-03 PT PT121579577T patent/PT2497500T/en unknown
- 2007-10-03 EP EP12157957.7A patent/EP2497500B1/en active Active
- 2007-10-03 PT PT78392164T patent/PT2066791E/en unknown
- 2007-10-03 PT PT161849666T patent/PT3146982T/en unknown
- 2007-10-03 PL PL07839216T patent/PL2066791T3/en unknown
- 2007-10-03 EP EP16184966.6A patent/EP3146982B1/en active Active
- 2007-10-03 HU HUE16184966A patent/HUE046709T2/en unknown
- 2007-10-03 AR ARP070104379A patent/AR063113A1/en active IP Right Grant
- 2007-10-03 CN CNA200780038545XA patent/CN101528922A/en active Pending
- 2007-10-03 DK DK07839216.4T patent/DK2066791T3/en active
2009
- 2009-04-03 US US12/417,910 patent/US9890394B2/en active Active
2016
- 2016-12-20 CY CY20161101325T patent/CY1118477T1/en unknown
2017
- 2017-12-29 US US15/858,303 patent/US10920245B2/en active Active

Also Published As

Publication number	Publication date
US10920245B2 (en)	2021-02-16
US20180230490A1 (en)	2018-08-16
EP2066791B1 (en)	2012-09-12
EP3146982A1 (en)	2017-03-29
LT3146982T (en)	2019-11-11
ES2391789T3 (en)	2012-11-29
EP3632472A1 (en)	2020-04-08
EP3146982B1 (en)	2019-08-21
PL2066791T3 (en)	2013-02-28
PT2497500T (en)	2016-11-23
HUE046709T2 (en)	2020-03-30
EP2066791A4 (en)	2010-11-03
HUE031182T2 (en)	2017-06-28
AR063113A1 (en)	2008-12-30
PT2066791E (en)	2012-12-05
DK2066791T3 (en)	2012-10-15
CN101528922A (en)	2009-09-09
EP2497500B1 (en)	2016-09-28
CN105770910A (en)	2016-07-20
ES2757723T3 (en)	2020-04-30
EP2066791A2 (en)	2009-06-10
PT3146982T (en)	2019-10-31
WO2008042420A2 (en)	2008-04-10
EP2497500A1 (en)	2012-09-12
PL2497500T3 (en)	2017-07-31
DK2497500T3 (en)	2017-01-02
ES2607060T3 (en)	2017-03-29
US9890394B2 (en)	2018-02-13
WO2008042420A3 (en)	2008-07-03
LT2497500T (en)	2016-12-27
SI3146982T1 (en)	2019-11-29
SI2066791T1 (en)	2013-01-31
CN108853517A (en)	2018-11-23
CY1118477T1 (en)	2017-07-12
US20090286857A1 (en)	2009-11-19

Publication	Publication Date	Title
LT2497500T (en)	2016-12-27	Gene therapy for spinal muscular atrophy
GB0620400D0 (en)	2006-11-22	Spinal implant
EP2094350A4 (en)	2010-07-14	Grouped leads for spinal stimulation
ZA200807936B (en)	2009-12-30	Compounds for the treatment of spinal muscular atrophy and other uses
IL197316A0 (en)	2009-12-24	Buprenophine-wafer for drug substitution therapy
EP2078536A4 (en)	2015-02-18	Medical apparatus
IL196559A0 (en)	2011-08-01	Combination therapy
GB0602178D0 (en)	2006-03-15	Therapeutic treatment
ZA200901376B (en)	2015-12-23	Trauma therapy
EP2081520A4 (en)	2012-08-01	Medical implants
IL196556A0 (en)	2009-11-18	Combination therapy
EP2180855A4 (en)	2014-09-03	Spinal correction apparatus
EP2054061A4 (en)	2009-09-02	Combination therapy
GB0618930D0 (en)	2006-11-08	Medical implant
GB0624200D0 (en)	2007-01-10	Wheelchairs
EP2099389A4 (en)	2013-01-09	Implantable spinal disk
ZA200902203B (en)	2010-08-25	Combination therapy
GB0608655D0 (en)	2006-06-14	Therapeutic Treatment
EP2022454A4 (en)	2010-08-04	Wheelchair
GB0602768D0 (en)	2006-03-22	Treatment of muscular dystrophy
GB0610909D0 (en)	2006-07-12	Therapeutic treatment
GB0617299D0 (en)	2006-10-11	Compounds for imaging and therapy
GB0607153D0 (en)	2006-05-17	Therapeutic Method
GB0602767D0 (en)	2006-03-22	Treatment of muscular dystrophy
IL255035A0 (en)	2017-12-31	Gene therapy for spinal cord disorders